Summary and Direction Forward
The unintended consequences of well intentioned actions produced a
situation in which children were therapeutic orphans, often denied the
benefits of research and evidence in guiding therapy. While there has
been considerable progress over the past two decades in addressing this
issue, much remains to be done, notably as the next generation of
therapeutics in the form of biologics, cell therapy and genetic
engineering evolve. Fortunately there is will and energy, notably among
the next generation of child health care providers and researchers, to
address and surmount these challenges in pursuit of the common goal of
effective and safe therapy for children everywhere.